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Latest News: Research

PTC Therapeutics Present Interim Data from FIREFISH Clinical Trial

21 June 2018 / Posted in: Research

PTC Therapeutics recently presented preliminary data from the FIREFISH Phase II clinical trial of their splice-modifying drug, RG7916, at the 2018 Cure SMA conference in Dallas, USA.

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Cytokinetics Release CK-2127107 Phase II Clinical Trial Data

20 June 2018 / Posted in: Research

Cytokinetics presented data at the 2018 Cure SMA conference from the Phase II clinical trial with SMA patients of its drug CK-2127107, which has been renamed reldesemtiv.

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Cabinet Secretary for Health and Sport in Scotland Outlines New Pathway For Medicines

19 June 2018 / Posted in: Research

A statement came out yesterday that explains how patients with rare diseases in Scotland could get faster access to new treatments following a revised decision-making process. The Scottish Government is introducing a new definition of 'ultra-orphan medicines' that can treat rare conditions.

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Latest Updates on Global Access to Nusinersen

18 June 2018 / Posted in: Research

We have asked Biogen to keep us updated with this latest information as we all continue to advocate for broad access in the UK.

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Roche Update: SUNFISH Trial Not Going Ahead In The UK

14 June 2018 / Posted in: Research

We have received the following update from Roche about the SUNFISH study, investigating RG7916 in people with SMA Types 2 or 3, which will now not be recruiting in the UK.

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First Cohort Dosing Completed in SRK-015 Phase 1 Clinical Trial

08 June 2018 / Posted in: Research

Pharmaceutical company Scholar Rock has announced that dosing of the first cohort has been completed in a Phase I trial of the experimental drug, SRK-015.

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Roche Community Update: Olesoxime

30 May 2018 / Posted in: Research

Due to negative long-term effects, Roche have decided to halt further development of their drug olesoxime. They have provided this community statement.

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Registration for the Public Gallery for the 27th June NICE Committee meeting

25 May 2018 / Posted in: Research

This opens on 30th May and closes 13th June. The meeting will discuss access to nusinersen and be held in Manchester. We understand that there are only 30 places available.

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Biogen's Response to Charities' Letter

23 May 2018 / Posted in: Research

On 16th May, the SMA charities wrote to leading managers of Biogen urging them to review their pricing and resubmit their application with the aim of Spinraza access being extended to those with SMA Types 2 and 3. Read Biogen's response.

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New International Standards of Care for SMA

22 May 2018 / Posted in: Research

You can now read the new recommendations as to what assessments and interventions families should expect to find in any neuromuscular centre. A family-friendly version is being worked on now.

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